The Company expects to submit a New Drug Application (NDA) for OC-01 nasal spray in the second half of 2020.

Positive topline data were announced from the phase 3 ONSET-2 study of OC-01 (varenicline; Oyster Point Pharma) nasal spray for the treatment of the signs and symptoms of dry eye disease.

OC-01 is a highly selective nicotinic acetylcholine receptor that stimulates natural tear film production by activating the trigeminal parasympathetic pathway in the nasal cavity.

The multicenter, randomized, double-masked, vehicle-controlled clinical study evaluated the efficacy and safety of OC-01 nasal spray in 785 patients aged ≥22 years for the treatment of the signs and symptoms of dry eye disease. Patients were randomized to receive OC-01 0.6mg/mL, 1.2mg/mL, or control twice daily for 4 weeks. The primary end point was the proportion of patients who achieved ≥10mm improvement in Schirmer’s Test Score from baseline to day 28. A key secondary end point was the mean change in Eye Dryness Score, as assessed on a visual analogue scale at day 28.

Results showed that the study met the primary end point with a significantly greater proportion of patients gaining ≥10mm improvement in Schirmer’s score when treated with OC-01 0.6mg/mL (44%; P <.0001) and 1.2mg/mL (47%; P <.0001) vs control (26%). Moreover, the mean change from baseline in Schirmer’s score was 11.0mm in the 0.6mg/mL arm and 11.2mm in the 1.2mg/mL arm vs 5.9mm in the control arm at week 4.

Regarding safety, the most common treatment-emergent adverse event was sneezing, which was transient and mild in severity. There were no reports of serious adverse events related to nasal administration.

“This novel treatment approach for dry eye disease is a much needed addition to the dry eye treatment armamentarium,” said Dr. Preeya Gupta, Associate Professor of Ophthalmology at Duke University School of Medicine and member of Oyster Point Pharma’s medical advisory board. “The ONSET-2 study results represent outcomes from a broad population of dry eye patients and we believe they should translate to treating patients in the clinic.”

In a statement, the Company noted that the statistical power for assessing the primary end point was impacted by a decrease in sample size, as assessments were not conducted due to the COVID-19 pandemic.

Oyster Point expects to submit a New Drug Application (NDA) for OC-01 nasal spray in the second half of 2020.